Drug Profile

ACTX 401

Alternative Names: ACTX-401

Latest Information Update: 25 Nov 2025

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At a glance

Originator Alcyone Lifesciences
Developer Alcyone Therapeutics
Class Gene therapies
Mechanism of Action Gene transference

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity No

Highest Development Phases

Phase I/II Spinal muscular atrophy
Preclinical Charcot-Marie-Tooth disease

Most Recent Events

14 Nov 2025 Alcyone Therapeutics has been acquired by Biogen
29 Sep 2022 Preclinical trials in Charcot-Marie-Tooth disease in USA (Parenteral) (Alcyone Therapeutics pipeline, September 2022)
18 May 2022 US FDA approves IND application for ACTX 401 in Spinal muscular atrophy

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